A mutation-independent approach via transcriptional upregulation of a disease modifier gene rescues muscular dystrophy in vivo

Dwi U. Kemaladewi; Prabhpreet S. Bassi; Steven Erwood; Dhekra Al-Basha; Kinga I. Gawlik; Kyle Lindsay; Ella Hyatt; Rebekah Kember; Kara M. Place; Ryan M. Marks; Madeleine Durbeej; Steven A. Prescott; Evgueni A. Ivakine; Ronald D. Cohn

doi:10.1101/286500

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A mutation-independent approach via transcriptional upregulation of a disease modifier gene rescues muscular dystrophy in vivo

View ORCID ProfileDwi U. Kemaladewi, Prabhpreet S. Bassi, Steven Erwood, Dhekra Al-Basha, Kinga I. Gawlik, Kyle Lindsay, Ella Hyatt, Rebekah Kember, Kara M. Place, Ryan M. Marks, View ORCID ProfileMadeleine Durbeej, Steven A. Prescott, Evgueni A. Ivakine, Ronald D. Cohn

doi: https://doi.org/10.1101/286500

Dwi U. Kemaladewi

1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Canada

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Prabhpreet S. Bassi

1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Canada

2Department of Molecular Genetics, University of Toronto, Canada

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Steven Erwood

1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Canada

2Department of Molecular Genetics, University of Toronto, Canada

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Dhekra Al-Basha

3Program in Neuroscience and Mental Health, the Hospital for Sick Children Research Institute, Toronto, Canada

4Department of Physiology, University of Toronto, Canada

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Kinga I. Gawlik

5Unit of Muscle Biology, Department of Experimental Medical Science, Lund University, Lund, Sweden

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Kyle Lindsay

1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Canada

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Ella Hyatt

1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Canada

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Rebekah Kember

1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Canada

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Kara M. Place

1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Canada

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Ryan M. Marks

1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Canada

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Madeleine Durbeej

5Unit of Muscle Biology, Department of Experimental Medical Science, Lund University, Lund, Sweden

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ORCID record for Madeleine Durbeej

Steven A. Prescott

3Program in Neuroscience and Mental Health, the Hospital for Sick Children Research Institute, Toronto, Canada

4Department of Physiology, University of Toronto, Canada

6Institute of Biomaterials and Biomedical Engineering, University of Toronto, Canada

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Evgueni A. Ivakine

1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Canada

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Ronald D. Cohn

1Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, Canada

2Department of Molecular Genetics, University of Toronto, Canada

7Department of Pediatrics, the Hospital for Sick Children, Toronto, Canada

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Posted November 11, 2018.

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A mutation-independent approach via transcriptional upregulation of a disease modifier gene rescues muscular dystrophy in vivo

Dwi U. Kemaladewi, Prabhpreet S. Bassi, Steven Erwood, Dhekra Al-Basha, Kinga I. Gawlik, Kyle Lindsay, Ella Hyatt, Rebekah Kember, Kara M. Place, Ryan M. Marks, Madeleine Durbeej, Steven A. Prescott, Evgueni A. Ivakine, Ronald D. Cohn

bioRxiv 286500; doi: https://doi.org/10.1101/286500

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